DK1916258T3 - Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar - Google Patents

Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar

Info

Publication number
DK1916258T3
DK1916258T3 DK07022775.6T DK07022775T DK1916258T3 DK 1916258 T3 DK1916258 T3 DK 1916258T3 DK 07022775 T DK07022775 T DK 07022775T DK 1916258 T3 DK1916258 T3 DK 1916258T3
Authority
DK
Denmark
Prior art keywords
sequence
base pairs
raav vector
raav
heterologous sequence
Prior art date
Application number
DK07022775.6T
Other languages
English (en)
Inventor
Barrie Carter
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=22575432&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK1916258(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Application granted granted Critical
Publication of DK1916258T3 publication Critical patent/DK1916258T3/da

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/191—Tumor necrosis factors [TNF], e.g. lymphotoxin [LT], i.e. TNF-beta
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70578—NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/06—Preparation of peptides or proteins produced by the hydrolysis of a peptide bond, e.g. hydrolysate products
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
DK07022775.6T 1999-08-09 2000-08-08 Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar DK1916258T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US16008099P 1999-08-09 1999-08-09
EP00953892A EP1204739B1 (en) 1999-08-09 2000-08-08 Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs

Publications (1)

Publication Number Publication Date
DK1916258T3 true DK1916258T3 (da) 2014-07-28

Family

ID=22575432

Family Applications (2)

Application Number Title Priority Date Filing Date
DK00953892T DK1204739T3 (da) 1999-08-09 2000-08-08 Forögelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar
DK07022775.6T DK1916258T3 (da) 1999-08-09 2000-08-08 Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DK00953892T DK1204739T3 (da) 1999-08-09 2000-08-08 Forögelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar

Country Status (12)

Country Link
US (8) US6596535B1 (da)
EP (2) EP2369002A1 (da)
JP (2) JP4827353B2 (da)
AT (1) ATE403715T1 (da)
AU (1) AU781958C (da)
CA (1) CA2379166C (da)
CY (1) CY1107432T1 (da)
DE (1) DE60039766D1 (da)
DK (2) DK1204739T3 (da)
ES (2) ES2478635T3 (da)
PT (2) PT1916258E (da)
WO (1) WO2001011034A2 (da)

Families Citing this family (313)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6989264B2 (en) * 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6893865B1 (en) * 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
EP2369002A1 (en) * 1999-08-09 2011-09-28 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
ATE318923T1 (de) * 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20040023390A1 (en) * 2002-08-05 2004-02-05 Davidson Beverly L. SiRNA-mediated gene silencing with viral vectors
US20040241854A1 (en) * 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
DK1664315T3 (da) * 2003-09-01 2012-07-09 Amc Amsterdam AAV-vektorer til in vivo genterapi af rheumatoid arthritis
US8529885B2 (en) 2003-09-01 2013-09-10 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
FR2874384B1 (fr) * 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
AU2005302408B2 (en) 2004-10-28 2012-08-09 The Government of the United States of America as represented by the Department of Veterans Affairs Peripherally delivered glutamic acid decarboxylase gene therapy for spinal cord injury pain
AU2006344750A1 (en) * 2006-06-15 2007-12-27 Targeted Genetics Corporation Methods for treating target joints in inflammatory arthritis using AAV vectors encoding a TNF antagonist
US8192441B2 (en) * 2008-10-03 2012-06-05 Howmedica Osteonics Corp. High tibial osteotomy instrumentation
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
KR20130040844A (ko) 2010-03-29 2013-04-24 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 약학적으로 유발된 전이유전자 제거 시스템
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
CN103491770B (zh) 2010-11-05 2016-06-08 斯坦福大学托管董事会 稳定阶跃函数视蛋白及其使用方法
CN106376525A (zh) 2010-11-05 2017-02-08 斯坦福大学托管董事会 记忆功能的控制和表征
EP3225108A1 (en) 2010-11-05 2017-10-04 The Board of Trustees of The Leland Stanford Junior University Optogenetic control of reward-related behaviors
CA2816971A1 (en) 2010-11-05 2012-05-10 The Board Of Trustees Of The Leland Stanford Junior University Light-activated chimeric opsins and methods of using the same
US20120263754A1 (en) 2011-02-15 2012-10-18 Immune Design Corp. Methods for Enhancing Immunogen Specific Immune Responses by Vectored Vaccines
AU2012243039B2 (en) 2011-04-08 2017-07-13 Immune Design Corp. Immunogenic compositions and methods of using the compositions for inducing humoral and cellular immune responses
US10352936B2 (en) 2011-04-14 2019-07-16 Apoplogic Pharmaceuticals, Inc. Use of tumor Fas expression to determine response to anti-cancer therapy
WO2012145509A2 (en) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors, and viruses
EP3524676A1 (en) 2011-12-16 2019-08-14 The Board of Trustees of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
EP2817068B1 (en) 2012-02-21 2017-04-12 The Board of Trustees of the Leland Stanford Junior University Compositions for treating neurogenic disorders of the pelvic floor
PT2850431T (pt) 2012-05-16 2018-07-23 Immune Design Corp Vacinas para hsv-2
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
BR112015009746A2 (pt) 2012-11-05 2017-08-15 Genzyme Corp Agente que aumenta atividade de glicocerebrosidase em mamíferos
EP3659518A1 (en) 2012-11-21 2020-06-03 Circuit Therapeutics, Inc. System and method for optogenetic therapy
SI2968461T1 (sl) 2013-03-13 2023-01-31 Genzyme Corporation Fuzijski proteini, ki vsebujejo vezavna dela PDGF in VEGF in postopek njihove uporabe
JP6591956B2 (ja) 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｍｐｓ１を治療するための組成物および方法
CA2906756A1 (en) 2013-03-15 2014-09-18 The Board Of Trustees Of The Leland Stanford Junior University Optogenetic control of behavioral state
CN105682674A (zh) 2013-04-17 2016-06-15 建新公司 用于治疗和预防黄斑变性的组合物和方法
JP6549559B2 (ja) 2013-04-29 2019-07-24 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 標的細胞における活動電位の光遺伝学的調節のための装置、システム及び方法
BR112015027336A2 (pt) 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
CN112516179A (zh) 2013-06-14 2021-03-19 普赛奥克苏斯治疗公司 用于b型腺病毒的给药方案及制剂
CN105829538A (zh) 2013-08-14 2016-08-03 小利兰·斯坦福大学托管委员会 用于控制疼痛的组合物和方法
SG11201602887QA (en) 2013-10-25 2016-05-30 Psioxus Therapeutics Ltd Oncolytic adenoviruses armed with heterologous genes
BR112016017817A2 (pt) 2014-02-06 2017-10-10 Genzyme Corp composições e métodos para o tratamento e a prevenção de degeneração macular
WO2015123496A1 (en) 2014-02-14 2015-08-20 Immune Design Corp. Immunotherapy of cancer through combination of local and systemic immune stimulation
JP6822841B2 (ja) 2014-03-09 2021-01-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物
WO2015141499A1 (ja) * 2014-03-20 2015-09-24 ヤンマー株式会社 エンジン装置及びこれを搭載した定置型作業機
DK3628334T5 (da) 2014-03-21 2024-09-02 Genzyme Corp Genterapi til behandling af retinitis pigmentosa
EP3122761B1 (en) 2014-03-28 2019-03-06 The Board of Trustees of The Leland Stanford Junior University Engineered light-activated anion channel proteins and methods of use thereof
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
CA2946392A1 (en) 2014-04-25 2015-10-29 James M. Wilson Ldlr variants and their use in compositions for reducing cholesterol levels
EP3137497B2 (en) 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
MY186389A (en) 2014-05-13 2021-07-22 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
KR20170032373A (ko) 2014-07-15 2017-03-22 이뮨 디자인 코포레이션 Tlr4 작용제 보조제 및 렌티바이러스 벡터를 이용한 프라임-부스트 요법
EP4012035B1 (en) 2014-09-16 2024-11-06 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
WO2016041588A1 (en) 2014-09-16 2016-03-24 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
WO2016044478A1 (en) 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
EP3247793A1 (en) 2015-01-20 2017-11-29 Genzyme Corporation Analytical ultracentrifugation for characterization of recombinant viral particles
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
CN116019934A (zh) 2015-02-10 2023-04-28 建新公司 病毒颗粒至纹状体和皮质的增强递送
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
JP6851319B2 (ja) 2015-04-27 2021-03-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
EA201792500A1 (ru) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Aav-опосредованная экспрессия антител против гриппа и способы их использования
RU2021132397A (ru) 2015-05-16 2022-02-24 Джензим Корпорейшн Генное редактирование глубоких интронных мутаций
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
US12221615B2 (en) 2015-08-06 2025-02-11 The Trustees Of The University Of Pennsylvania GLP-1 and use thereof in compositions for treating metabolic diseases
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
CA2995733A1 (en) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo for treating companion animals
EP3359663A4 (en) 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
JP2019501115A (ja) 2015-10-28 2019-01-17 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 遺伝子治療のためのアデノ随伴ウイルスベクターのくも膜下腔内投与
CA3008142A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
AU2016370590B2 (en) 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
WO2017106202A2 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
EA201891022A1 (ru) 2015-12-17 2019-01-31 Псайоксус Терапьютикс Лимитед Вирус, кодирующий антитело к комплексу tcr или фрагмент указанного антитела
DK3402530T3 (da) * 2016-01-13 2020-11-23 Genzyme Corp Rekombinante aav-vektorer, der udtrykker knoglebeskyttende gener, herunder has2 og lubricin, som er anvendelige til behandling af osteoarthritis og relaterede ledtilstande hos pattedyr
AU2017215211C1 (en) 2016-02-03 2023-11-16 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
US12070510B2 (en) 2016-02-05 2024-08-27 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
IL310729A (en) 2016-04-15 2024-04-01 Alpine Immune Sciences Inc Immunomodulatory proteins that are variants of CD80 and their uses
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
MX2018012472A (es) 2016-04-15 2019-08-12 Alpine Immune Sciences Inc Proteinas inmunomoduladoras variantes de ligando icos y sus usos.
AU2017250298B2 (en) 2016-04-15 2024-10-03 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
WO2017180936A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
US11401527B2 (en) 2016-04-17 2022-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
KR102799528B1 (ko) 2016-07-08 2025-04-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
US11471488B2 (en) 2016-07-28 2022-10-18 Alpine Immune Sciences, Inc. CD155 variant immunomodulatory proteins and uses thereof
EP3491013A1 (en) 2016-07-28 2019-06-05 Alpine Immune Sciences, Inc. Cd155 variant immunomodulatory proteins and uses thereof
WO2018022945A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd112 variant immunomodulatory proteins and uses thereof
SG10201913002QA (en) 2016-08-15 2020-03-30 Genzyme Corp Methods for detecting aav
MY197324A (en) 2016-08-29 2023-06-13 Akamis Bio Ltd Adenovirus armed with bispecific t cell activator
GB201713765D0 (en) 2017-08-28 2017-10-11 Psioxus Therapeutics Ltd Modified adenovirus
WO2018132747A1 (en) 2017-01-13 2018-07-19 University Of Iowa Research Foundation Bocaparvovirus small noncoding rna and uses thereof
MA47436A (fr) 2017-01-31 2019-12-11 Regenxbio Inc Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua)
KR20190118163A (ko) 2017-02-20 2019-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
BR112019017697A2 (pt) 2017-02-28 2020-04-07 The Trustees Of The University Of Pennsylvania vacinas contra influenza baseadas em vetores de vírus adenoassociado (aav)
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
MX2019010275A (es) 2017-02-28 2019-10-15 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este.
KR20230093072A (ko) 2017-03-01 2023-06-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 안구 장애에 대한 유전자 치료
IL320149A (en) 2017-03-16 2025-06-01 Alpine Immune Sciences Inc Pd-l1 variant immunomodulatory proteins and uses thereof
JP2020511144A (ja) 2017-03-16 2020-04-16 アルパイン イミューン サイエンシズ インコーポレイテッド Ｐｄ−ｌ２バリアント免疫調節タンパク質及びその使用
EP3596114A2 (en) 2017-03-16 2020-01-22 Alpine Immune Sciences, Inc. Cd80 variant immunomodulatory proteins and uses thereof
BR112019019813A2 (pt) 2017-03-30 2020-04-22 The University Of Queensland moléculas quiméricas e usos das mesmas
AU2018253303B2 (en) 2017-04-14 2024-11-21 Regenxbio Inc. Treatment of mucopolysaccharidosis II with recombinant human iduronate-2 sulfatase (IDS) produced by human neural or glial cells
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
BR112019023303A2 (pt) 2017-05-11 2020-06-16 The Trustees Of The University Of Pennsylvania Terapia de gene para lipofuscinoses ceroides neuronais
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (ｆｇｆ21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
CA3098592A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
JP7394628B2 (ja) * 2017-06-01 2023-12-08 アカミス バイオ リミテッド 腫瘍溶解性ウイルスおよび方法
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
EP3676385A1 (en) 2017-07-06 2020-07-08 The Trustees of The University of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
EP3687561A4 (en) 2017-09-01 2021-06-09 The Australian National University "immunoregulatory molecules and uses therefor"
JP7449223B2 (ja) 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉ型を治療するための遺伝子療法
IL273394B2 (en) 2017-09-22 2025-04-01 Genzyme Corp RNAI variant
JP7511469B2 (ja) 2017-09-27 2024-07-05 イノザイム ファーマ， インコーポレイテッド 組換えエクトヌクレオチドピロホスファターゼ／ホスホジエステラーゼ（ｎｐｐ１）を使用して心血管機能を改善し、心血管疾患を処置するための方法。
WO2019074983A1 (en) 2017-10-10 2019-04-18 Alpine Immune Sciences, Inc. CTLA-4 VARIANT IMMUNOMODULATORY PROTEINS AND USES THEREOF
AU2018350990A1 (en) 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
JP7282760B2 (ja) 2017-10-18 2023-05-29 アルパイン イミューン サイエンシズ インコーポレイテッド バリアントｉｃｏｓリガンド免疫調節タンパク質ならびに関連する組成物および方法
AU2018350992A1 (en) 2017-10-18 2020-05-21 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
EA202091352A1 (ru) 2017-11-30 2020-08-27 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Генотерапия мукополисахаридоза iiib
AU2018375164B2 (en) 2017-11-30 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for Mucopolysaccharidosis IIIA
WO2019136179A1 (en) 2018-01-03 2019-07-11 Alpine Immune Sciences, Inc. Multi-domain immunomodulatory proteins and methods of use thereof
GB201801614D0 (en) 2018-01-31 2018-03-14 Psioxus Therapeutics Ltd Formulation
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
AU2019228504B2 (en) 2018-02-27 2025-06-26 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CA3099958A1 (en) 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
US12365865B2 (en) 2018-04-29 2025-07-22 Regenxbio Inc. Scalable clarification process for recombinant AAV production
EP3788165A1 (en) 2018-04-29 2021-03-10 REGENXBIO Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
WO2019241535A2 (en) 2018-06-14 2019-12-19 Regenxbio Inc. Anion exchange chromatography for recombinant aav production
US12065476B2 (en) 2018-06-15 2024-08-20 Alpine Immune Sciences, Inc. PD-1 variant immunomodulatory proteins and uses thereof
CA3108526A1 (en) 2018-08-03 2020-02-06 Genzyme Corporation Variant rnai against alpha-synuclein
US12612644B2 (en) 2018-08-10 2026-04-28 Regenxbio Inc. Scalable method for recombinant AAV production
CN110857440B (zh) 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
KR20210089146A (ko) 2018-09-19 2021-07-15 알파인 이뮨 사이언시즈, 인코포레이티드 변이체 cd80 단백질 및 관련 구축물의 방법 및 용도
EP4461814A3 (en) 2018-10-12 2025-02-26 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
US20210324483A1 (en) 2018-10-15 2021-10-21 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
CN111118016B (zh) 2018-10-30 2022-04-15 上海朗昇生物科技有限公司 治疗视网膜色素变性疾病的基因治疗载体
US12370240B2 (en) 2018-11-28 2025-07-29 Genascence Corporation Methods and compositions for treating osteoarthritis
JP7713886B2 (ja) 2018-11-30 2025-07-28 アルパイン イミューン サイエンシズ インコーポレイテッド Ｃｄ８６バリアント免疫調節タンパク質およびその使用
WO2020142714A1 (en) 2019-01-04 2020-07-09 Exonics Therapeutics, Inc. Aav expression cassette and aav vectors comprising the same
SG11202108480UA (en) 2019-02-26 2021-09-29 Univ Pennsylvania Compositions useful in treatment of krabbe disease
JP7637060B2 (ja) 2019-03-04 2025-02-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ａｋｔ経路を標的とする神経保護遺伝子療法
CN111718418B (zh) 2019-03-19 2021-08-27 华东师范大学 一种增强基因编辑的融合蛋白及其应用
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
WO2020205889A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
MX2021011889A (es) 2019-04-03 2021-10-26 Regenxbio Inc Tratamiento genico para patologias oculares.
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
US12565645B2 (en) 2019-04-11 2026-03-03 Regenxbio Inc. Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions
KR20220035032A (ko) 2019-04-17 2022-03-21 알파인 이뮨 사이언시즈, 인코포레이티드 변이체 icos 리간드(icosl) 융합 단백질의 방법 및 용도
WO2020214929A1 (en) 2019-04-19 2020-10-22 Regenxbio Inc. Adeno-associated virus vector formulations and methods
TW202106708A (zh) 2019-04-24 2021-02-16 美商銳進科斯生物股份有限公司 全人類轉譯後修飾之抗體治療劑
AU2020270421A1 (en) 2019-05-03 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
CN112180094A (zh) 2019-07-04 2021-01-05 上海东慈生物科技有限公司 Dkk1抑制剂在预防和/或治疗肿瘤恶病质与糖尿病伴随疾病中的应用
EP3914720B9 (en) 2019-07-10 2023-09-27 Masonic Medical Research Laboratory Vgll4 with ucp-1 cis-regulatory element and method of use thereof
EP3997225A1 (en) 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
EP4004214A1 (en) 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
US20220273726A1 (en) 2019-08-16 2022-09-01 Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences Treatment of neuronal diseases
BR112022003811A2 (pt) 2019-08-26 2022-11-16 Regenxbio Inc Tratamento de retinopatia diabética com fab anti vegf totalmente humano modificado pós tradução
CN114728049A (zh) 2019-10-07 2022-07-08 再生生物股份有限公司 腺相关病毒载体药物组合物和方法
EP4045093A4 (en) 2019-10-14 2024-02-07 Duke University Compositions comprising novel prokaryotic sodium channels and associated methods
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
WO2021099394A1 (en) 2019-11-19 2021-05-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides and their use for the treatment of cancer
US20230270886A1 (en) 2019-11-28 2023-08-31 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
EP4093428A1 (en) 2020-01-22 2022-11-30 RegenxBio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
WO2021154956A1 (en) 2020-01-29 2021-08-05 Regenxbio Inc. Treatment of mucopolysaccharidosis iva
TW202142695A (zh) 2020-01-29 2021-11-16 美商銳進科斯生物股份有限公司 用人類神經或神經膠質細胞產生之重組人類艾杜糖醛酸—２—硫酸酯酶（ｉｄｓ）對ｉｉ型黏多糖病之治療
GB202004498D0 (en) 2020-03-27 2020-05-13 Ucl Business Ltd Activity-dependent gene therapy for neurological disorders
JP2023528139A (ja) 2020-03-30 2023-07-04 ウーハン ニューロフス バイオテクノロジー リミテッド カンパニー ヒト核因子ｅ２関連因子２の発現ベクター及び発現ベクターの適用
WO2021226551A1 (en) 2020-05-08 2021-11-11 Alpine Immune Sciences, Inc. April and baff inhibitory immunomodulatory proteins and methods of use thereof
US20230210941A1 (en) 2020-05-12 2023-07-06 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
CA3177407A1 (en) 2020-05-12 2021-11-18 James M. Wilson Compositions for drg-specific reduction of transgene expression
JP2023531451A (ja) 2020-06-17 2023-07-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 遺伝子療法患者の治療のための組成物及び方法
IL299771A (en) 2020-07-10 2023-03-01 Inst Nat Sante Rech Med Methods and compounds for the treatment of epilepsy
GB202010981D0 (en) 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
US20230265456A1 (en) 2020-08-10 2023-08-24 Fundacion Para La Investigacion Medica Aplicada Gene therapy vector expressing cyp27a1 for the treatment of cerebrotendinous xanthomatosis
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
BR112023003310A2 (pt) 2020-08-24 2023-05-02 Univ Pennsylvania Vetores virais que codificam fusões de agonistas do receptor de glp-1 e usos dos mesmos no tratamento de doenças metabólicas
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
US12173308B2 (en) 2020-10-01 2024-12-24 Genzyme Corporation Human PAH expression cassette for treatment of PKU by liver-directed gene replacement therapy
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
AU2021358048A1 (en) 2020-10-07 2023-05-25 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
US20230364206A1 (en) 2020-10-07 2023-11-16 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
CA3198368A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
AU2021356667A1 (en) 2020-10-07 2023-06-08 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
IL301947A (en) 2020-10-07 2023-06-01 Regenxbio Inc Formulations for suprachoroidal administration such as gel formulations
AU2021356684A1 (en) 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
JP2023547832A (ja) 2020-10-28 2023-11-14 リジェネックスバイオ インコーポレイテッド 眼の適応症に対するベクター化抗ＴＮＦ－α抗体
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
EP4237453A1 (en) 2020-10-29 2023-09-06 RegenxBio Inc. Vectorized tnf-alpha antagonists for ocular indications
CA3196499A1 (en) 2020-10-29 2022-05-05 James M. Wilson Aav capsids and compositions containing same
EP4236974A2 (en) 2020-10-29 2023-09-06 RegenxBio Inc. Vectorized factor xii antibodies and administration thereof
JP2024016295A (ja) * 2020-11-30 2024-02-07 タカラバイオ株式会社 二価の陽イオンを利用した非エンベロープウイルスの製造方法
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
JP2023554389A (ja) 2020-12-16 2023-12-27 リジェネクスバイオ インコーポレイテッド 組換えアデノ随伴ウイルスウイルス粒子を生成する方法
WO2022136616A1 (en) 2020-12-23 2022-06-30 Vivet Therapeutics Minimal bile acid inducible promoters for gene therapy
US20240309076A1 (en) 2020-12-29 2024-09-19 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
JP2024503895A (ja) 2021-01-21 2024-01-29 リジェネクスバイオ インコーポレイテッド 組換えポリペプチド及びウイルスの生成の改善
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
BR112023016075A2 (pt) 2021-02-10 2023-11-21 Regenxbio Inc Método para tratar hepatoesplenomegalia, método de distribuição de um raav que codifica hids, método para tratar um sujeito humano, método de tratamento de um sujeito humano, método para determinar a eficácia do tratamento, método para identificar um sujeito como tendo mps ii
CN117460832A (zh) 2021-03-22 2024-01-26 建新公司 空aav衣壳和完整aav衣壳的尺寸排阻色谱分析
MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
US20240216543A1 (en) 2021-04-26 2024-07-04 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2022235614A2 (en) 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
EP4333869A1 (en) 2021-05-07 2024-03-13 Alpine Immune Sciences, Inc. Methods of dosing and treatment with a taci-fc fusion immunomodulatory protein
US20240358857A1 (en) 2021-05-11 2024-10-31 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
EP4373528A2 (en) 2021-07-19 2024-05-29 New York University Adeno-associated viral vector compositions and methods of promoting muscle regeneration
US20240409955A1 (en) 2021-07-19 2024-12-12 New York University Auf1 combination therapies for treatment of muscle degenerative disease
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
TW202325845A (zh) 2021-10-02 2023-07-01 賓州大學委員會 新穎aav衣殼及含其之組成物
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
EP4413019A2 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
EP4413018A1 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023062652A1 (en) * 2021-10-13 2023-04-20 Haystackanalyt Ics Pvt. Ltd Systems and methods for secure genomic analysis using a specialized edge computing device
WO2023077092A1 (en) 2021-10-28 2023-05-04 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
US20250064974A1 (en) 2022-01-06 2025-02-27 Ucl Business Ltd Endogenous gene regulation to treat neurological disorders and diseases
TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
GB202201744D0 (en) 2022-02-10 2022-03-30 Ucl Business Ltd Treatment of acquired focal epilepsy
US20250243260A1 (en) 2022-03-07 2025-07-31 Alpine Immune Sciences, Inc. Immunomodulatory proteins of variant cd80 polypeptides, cell therapies and related methods and uses
WO2023178053A1 (en) 2022-03-13 2023-09-21 Regenxbio Inc. Modified muscle-specific promoters
EP4499154A1 (en) 2022-03-25 2025-02-05 REGENXBIO Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
CA3247507A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. PHARMACEUTICAL COMPOSITION COMPRISING A RECOMMENDED ADENO-ASSOCIATED VIRUS (AAV) VECTOR WITH AN EXPRESSION CASSETTE ENCODED BY A TRANSGENE FOR SUPRACHOROID ADMINISTRATION
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
IL316078A (en) 2022-04-06 2024-12-01 Genzyme Corp Targeted gene therapy for myotonic dystrophy DM-1
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
TW202404993A (zh) 2022-04-11 2024-02-01 美商特納亞治療股份有限公司 具經工程化蛋白殼之腺相關病毒
EP4508428A1 (en) 2022-04-12 2025-02-19 Genzyme Corporation Dendritic cell assay for innate immunogenicity to gene therapy agents
AU2023252915A1 (en) 2022-04-12 2024-11-28 Genzyme Corporation Use of irak4 modulators for gene therapy
JP2025513851A (ja) 2022-04-12 2025-04-30 ジェンザイム・コーポレーション 遺伝子治療のためのｉｒａｋ４モジュレーターの使用
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250249127A1 (en) 2022-04-14 2025-08-07 Regenxbio Inc. Gene therapy for treating an ocular disease
US20250277004A1 (en) 2022-04-18 2025-09-04 Regenxbio Inc. Hybrid aav capsids
WO2023215806A2 (en) 2022-05-03 2023-11-09 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2023215807A1 (en) 2022-05-03 2023-11-09 Regenxbio Inc. VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS
JP2025517628A (ja) 2022-05-06 2025-06-10 ノバルティス アーゲー 新規の組換えａａｖ ｖｐ２融合ポリペプチド
AU2023273809A1 (en) 2022-05-16 2025-01-02 Genzyme Corporation Methods of treating metachromatic leukodystrophy
AU2023273230A1 (en) 2022-05-17 2024-11-07 Centre Hospitalier Universitaire Vaudois Designed biosensors for enhanced t cell therapy
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
US20260021205A1 (en) 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
US20260055144A1 (en) 2022-08-24 2026-02-26 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
EP4593955A1 (en) 2022-09-30 2025-08-06 RegenxBio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
CN120344256A (zh) 2022-10-04 2025-07-18 高山免疫科学股份有限公司 突变的taci-fc融合蛋白用于在治疗自身抗体介导的疾病中的用途
JP2025534666A (ja) 2022-10-11 2025-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
CN117947040A (zh) 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
CN118576695A (zh) 2023-03-01 2024-09-03 上海三弹生物医药科技有限公司 一种治疗或改善恶病质的方法
EP4680741A2 (en) 2023-03-15 2026-01-21 RegenxBio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
CN121002180A (zh) 2023-04-05 2025-11-21 建新公司 Dm-1肌强直性营养不良的靶向基因疗法
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
AU2024271004A1 (en) 2023-05-16 2026-01-15 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
EP4724085A1 (en) 2023-06-12 2026-04-15 The Trustees of The University of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
CN116790615B (zh) 2023-07-11 2024-11-22 康霖生物科技（杭州）有限公司 一种用于过敏性疾病的基因治疗载体核酸构建体及其使用方法
WO2025016362A1 (zh) 2023-07-19 2025-01-23 中国科学院脑科学与智能技术卓越创新中心 基于脑内磷稳态促进剂的疗法
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025106874A1 (en) 2023-11-15 2025-05-22 Genzyme Corporation Methods of treating neurodegenerative disorders
US20250152747A1 (en) 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating gaucher disease and gba-parkinson’s disease
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
US20250197862A1 (en) 2023-12-15 2025-06-19 Genzyme Corporation ARTIFICIAL microRNAs TARGETING TAU
TW202547561A (zh) 2024-01-25 2025-12-16 美商健臻公司 基於ｆｕｓ向大腦遞送病毒顆粒之方法
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
TW202546230A (zh) 2024-01-26 2025-12-01 美商健臻公司 靶向ｓｎｃａ之人工ｍｉｃｒｏｒｎａ
WO2025160434A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease
WO2025189030A1 (en) 2024-03-07 2025-09-12 Stellaromics, Inc. Methods and systems for volumetric imaging
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025235643A1 (en) 2024-05-08 2025-11-13 Genzyme Corporation Profiling of gene therapy agents
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5605690A (en) 1989-09-05 1997-02-25 Immunex Corporation Methods of lowering active TNF-α levels in mammals using tumor necrosis factor receptor
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5869305A (en) * 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6686200B1 (en) * 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
ATE272123T1 (de) 1993-11-09 2004-08-15 Ohio Med College Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US6281010B1 (en) * 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US5677158A (en) * 1995-06-07 1997-10-14 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
CA2247099A1 (en) 1996-03-04 1997-09-12 Targeted Genetics Corporation Methods for transducing cells in blood vessels using recombinant aav vectors
US6541258B2 (en) 1996-12-18 2003-04-01 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
EP0946723A1 (en) 1996-12-18 1999-10-06 Targeted Genetics Corporation Recombinase-activatable aav packaging cassettes for use in the production of aav vectors
US6605429B1 (en) 1997-01-23 2003-08-12 Immusol, Incorporated Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
DK1944362T3 (da) 1997-09-05 2016-01-25 Genzyme Corp Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer
JP2001520047A (ja) 1997-10-21 2001-10-30 ターゲテッド ジェネティックス コーポレイション 組換えａａｖベクターと共に使用するための転写活性化された逆方向末端反復（ｉｔｒ）
AU758541B2 (en) 1997-10-21 2003-03-27 Targeted Genetics Corporation Amplifiable adeno-associated virus (AAV) packaging cassettes for the production of recombinant AAV vectors
DK1109892T3 (da) 1998-09-04 2009-02-09 Targeted Genetics Corp Fremgangsmåder til at frembringe höjtitrede, hjælpevirusfrie præparater af frie rekombinante AAV-vektorer
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
EP1180159B1 (en) 1999-05-28 2008-09-03 Targeted Genetics Corporation Methods and compositions for lowering the level of tumor necrosis factor (tnf) in tnf-associated disorders
EP2369002A1 (en) 1999-08-09 2011-09-28 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
ATE318923T1 (de) * 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
JP6143466B2 (ja) 2013-01-10 2017-06-07 株式会社ジャパンディスプレイ タッチパネル内蔵型液晶表示装置

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